Immunogenicity assessment strategy for a chemically modified therapeutic protein in clinical development

The clinical immunogenicity assessment for complex multidomain biological drugs is challenging due to multiple factors that must be taken into consideration. Here, we describe a strategy to overcome multiple bioanalytical challenges in order to assess anti-drug antibodies (ADA) for a novel and uniqu...

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Main Authors: Charlotte Hagman, Gaetan Chasseigne, Robert Nelson, Florian Anlauff, Mark Kagan, Allison B. Goldfine, Grzegorz Terszowski, Maria Jadhav
Format: Article
Language:English
Published: Frontiers Media S.A. 2024-11-01
Series:Frontiers in Immunology
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Online Access:https://www.frontiersin.org/articles/10.3389/fimmu.2024.1438251/full
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author Charlotte Hagman
Gaetan Chasseigne
Robert Nelson
Florian Anlauff
Mark Kagan
Allison B. Goldfine
Grzegorz Terszowski
Maria Jadhav
author_facet Charlotte Hagman
Gaetan Chasseigne
Robert Nelson
Florian Anlauff
Mark Kagan
Allison B. Goldfine
Grzegorz Terszowski
Maria Jadhav
author_sort Charlotte Hagman
collection DOAJ
description The clinical immunogenicity assessment for complex multidomain biological drugs is challenging due to multiple factors that must be taken into consideration. Here, we describe a strategy to overcome multiple bioanalytical challenges in order to assess anti-drug antibodies (ADA) for a novel and unique chemically modified protein therapeutic. A risk-centered approach was adopted to evaluate the immunogenic response to a modified version of human growth differentiation factor 15 (GDF15) connected to an albumin-binding fatty acid via a polyethylene glycol (PEG) linker. Key steps include monitoring anti-drug antibodies (ADAs), using a standard tiered approach of screening and confirmation. To deepen our understanding of ADA response, as a third tier of immunogenicity assessment, novel extensive characterization using a set of assays was developed, validated, and used routinely in clinical sample analysis. This characterization step included performance of titration, mapping of ADA response including anti-GDF15 and anti-PEG–fatty-acid antibody characterization, and assessment of the neutralizing anti-drug antibodies (NAbs) using cell-based assays for immunogenicity in parallel. The analytical methods were applied during two clinical trials involving both healthy volunteers and overweight or obese patients. We observed low incident rates for ADA and no ADAs against the PEG linker with fatty acid conjugation. In one of the clinical studies, we identified neutralizing ADAs. The proposed novel strategy of extensive characterization proved effective for monitoring the presence of ADAs and NAbs and can be used to support clinical development of a broad range of chemically modified proteins and multidomain biotherapeutics.
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spelling doaj-art-7b5a650d46c844bcaf6b7b4b75ba816c2024-11-11T06:10:19ZengFrontiers Media S.A.Frontiers in Immunology1664-32242024-11-011510.3389/fimmu.2024.14382511438251Immunogenicity assessment strategy for a chemically modified therapeutic protein in clinical developmentCharlotte Hagman0Gaetan Chasseigne1Robert Nelson2Florian Anlauff3Mark Kagan4Allison B. Goldfine5Grzegorz Terszowski6Maria Jadhav7Pharmacokinetic Sciences - Drug Disposition, Biomedical Research, Novartis, Basel, SwitzerlandPharmacokinetic Sciences - Drug Disposition, Biomedical Research, Novartis, Basel, SwitzerlandBioAgilytix Laboratories, Hamburg, GermanyBioAgilytix Laboratories, Hamburg, GermanyPharmacokinetic Sciences, Biomedical Research, Novartis, East Hanover, NJ, United StatesTranslational Medicine, Biomedical Research, Novartis, Cambridge, MA, United StatesPharmacokinetic Sciences - Drug Disposition, Biomedical Research, Novartis, Basel, SwitzerlandPharmacokinetic Sciences - Drug Disposition, Biomedical Research, Novartis, Cambridge, MA, United StatesThe clinical immunogenicity assessment for complex multidomain biological drugs is challenging due to multiple factors that must be taken into consideration. Here, we describe a strategy to overcome multiple bioanalytical challenges in order to assess anti-drug antibodies (ADA) for a novel and unique chemically modified protein therapeutic. A risk-centered approach was adopted to evaluate the immunogenic response to a modified version of human growth differentiation factor 15 (GDF15) connected to an albumin-binding fatty acid via a polyethylene glycol (PEG) linker. Key steps include monitoring anti-drug antibodies (ADAs), using a standard tiered approach of screening and confirmation. To deepen our understanding of ADA response, as a third tier of immunogenicity assessment, novel extensive characterization using a set of assays was developed, validated, and used routinely in clinical sample analysis. This characterization step included performance of titration, mapping of ADA response including anti-GDF15 and anti-PEG–fatty-acid antibody characterization, and assessment of the neutralizing anti-drug antibodies (NAbs) using cell-based assays for immunogenicity in parallel. The analytical methods were applied during two clinical trials involving both healthy volunteers and overweight or obese patients. We observed low incident rates for ADA and no ADAs against the PEG linker with fatty acid conjugation. In one of the clinical studies, we identified neutralizing ADAs. The proposed novel strategy of extensive characterization proved effective for monitoring the presence of ADAs and NAbs and can be used to support clinical development of a broad range of chemically modified proteins and multidomain biotherapeutics.https://www.frontiersin.org/articles/10.3389/fimmu.2024.1438251/fullimmunogenicityclinical developmenttherapeutic proteinschemical modificationendogenous counterpart
spellingShingle Charlotte Hagman
Gaetan Chasseigne
Robert Nelson
Florian Anlauff
Mark Kagan
Allison B. Goldfine
Grzegorz Terszowski
Maria Jadhav
Immunogenicity assessment strategy for a chemically modified therapeutic protein in clinical development
Frontiers in Immunology
immunogenicity
clinical development
therapeutic proteins
chemical modification
endogenous counterpart
title Immunogenicity assessment strategy for a chemically modified therapeutic protein in clinical development
title_full Immunogenicity assessment strategy for a chemically modified therapeutic protein in clinical development
title_fullStr Immunogenicity assessment strategy for a chemically modified therapeutic protein in clinical development
title_full_unstemmed Immunogenicity assessment strategy for a chemically modified therapeutic protein in clinical development
title_short Immunogenicity assessment strategy for a chemically modified therapeutic protein in clinical development
title_sort immunogenicity assessment strategy for a chemically modified therapeutic protein in clinical development
topic immunogenicity
clinical development
therapeutic proteins
chemical modification
endogenous counterpart
url https://www.frontiersin.org/articles/10.3389/fimmu.2024.1438251/full
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